JP2021534814A - アルファ−シヌクレインに対するバリアントＲＮＡｉ - Google Patents
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| US201862714616P | 2018-08-03 | 2018-08-03 | |
| US62/714,616 | 2018-08-03 | ||
| PCT/US2019/044924 WO2020028816A1 (en) | 2018-08-03 | 2019-08-02 | Variant rnai against alpha-synuclein |
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| JP2021534814A true JP2021534814A (ja) | 2021-12-16 |
| JPWO2020028816A5 JPWO2020028816A5 (es) | 2022-08-04 |
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| AR113134A1 (es) * | 2017-09-22 | 2020-01-29 | Genzyme Corp | Arni variante |
| AU2021238326A1 (en) * | 2020-03-18 | 2022-10-13 | University Of Massachusetts | Oligonucleotides for SNCA modulation |
| EP4359525A1 (en) * | 2021-06-21 | 2024-05-01 | uniQure biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| US20230365968A1 (en) * | 2022-04-06 | 2023-11-16 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012027713A2 (en) * | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
| JP2014501507A (ja) * | 2010-11-17 | 2014-01-23 | アイシス ファーマシューティカルズ， インコーポレーテッド | α−シヌクレイン発現の調節 |
| JP2018506304A (ja) * | 2015-02-10 | 2018-03-08 | ジェンザイム・コーポレーション | バリアントＲＮＡｉ |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| WO1998010088A1 (en) | 1996-09-06 | 1998-03-12 | Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JP4827353B2 (ja) | 1999-08-09 | 2011-11-30 | ターゲティッド ジェネティクス コーポレイション | 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大 |
| DE60117550T2 (de) | 2000-06-01 | 2006-12-07 | University Of North Carolina At Chapel Hill | Doppelsträngige parvovirus-vektoren |
| US20050137155A1 (en) * | 2001-05-18 | 2005-06-23 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA) |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| SG157224A1 (en) | 2001-11-13 | 2009-12-29 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| CA2691617C (en) | 2007-05-31 | 2020-07-21 | University Of Iowa Research Foundation | Reduction of off-target rna interference toxicity |
| GB0816778D0 (en) * | 2008-09-12 | 2008-10-22 | Isis Innovation | Gene silencing |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| SG10201601110VA (en) | 2011-02-17 | 2016-03-30 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| DK3137497T3 (da) | 2014-05-02 | 2021-07-12 | Genzyme Corp | Aav-vektorer til retinal- og cns-genterapi |
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Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012027713A2 (en) * | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
| JP2014501507A (ja) * | 2010-11-17 | 2014-01-23 | アイシス ファーマシューティカルズ， インコーポレーテッド | α−シヌクレイン発現の調節 |
| JP2018506304A (ja) * | 2015-02-10 | 2018-03-08 | ジェンザイム・コーポレーション | バリアントＲＮＡｉ |
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| EP3830265A1 (en) | 2021-06-09 |
| AR114540A1 (es) | 2020-09-16 |
| AU2019314529A1 (en) | 2021-03-25 |
| TW202022116A (zh) | 2020-06-16 |
| KR20210062627A (ko) | 2021-05-31 |
| BR112021001980A2 (pt) | 2021-05-04 |
| CO2021002637A2 (es) | 2021-07-30 |
| IL280581A (en) | 2021-03-25 |
| US20210309999A1 (en) | 2021-10-07 |
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| WO2020028816A1 (en) | 2020-02-06 |
| CN112805382A (zh) | 2021-05-14 |
| SG11202101072UA (en) | 2021-03-30 |
| MX2021001395A (es) | 2021-08-11 |
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